Spinocerebellar ataxia (SCA) is a group of inherited disorders characterized by cerebellar degeneration leading to imbalance, incoordination, speech difficulties and problems with walking. Recently, individual case reports have suggested that varenicline, a drug used in smoking cessation, produces substantial improvement in patients with several inherited ataxias. A modest response was noted in 5 patients with SCA, suggesting that it is potentially efficacious in this disorder as well. Although this agent is available for off-label use, the severe side effects noted with its use and the lack of long-term toxicity data demand that it be systematically assessed. The present study will test whether varenicline is safe and potentially efficacious in a heterogeneous cohort of adults with SCA.

Stable doses of all medications for 30 days prior to study entry and for the duration of the study.

Ability to ambulate with or without assistance.

Score of 10 or higher (worse) on the SARA total score.

Score of 3 or higher (worse) on the 'gait' subsection of the SARA rating scale.

Exclusion Criteria:

Any unstable illness or concomitant medical condition that, in the investigator's opinion, precludes participation in this study. This includes other disorders that may affect gait or balance (stroke, arthritis, etc).

Pregnancy or lactation.

Concurrent participation in another clinical study.

Patients with a history of substance abuse.

Patients who currently smoke or have smoked within the past 12 months.

Presence of psychosis, bipolar disorder, untreated depression (BDI greater than or equal to 21), or history of suicide attempt.

Concurrent treatment with any MAOIs, Wellbutrin, or nicotine patches.

Dementia or other psychiatric illness that prevents the patient from giving informed consent (Mini Mental Status Exam score less than 24).

Ataxia derived from any other cause than genetically-confirmed SCA (including but not limited to alcoholism, head injury, Multiple Sclerosis, olivo-ponto-cerebellar atrophy or multiple system atrophy).

Contacts and Locations

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study.
To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below.
For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00992771

Locations

United States, California

University of California - Los Angeles

Los Angeles, California, United States, 90095

United States, Florida

University of Florida

Gainesville, Florida, United States, 32610

University of South Florida

Tampa, Florida, United States, 33612

Sponsors and Collaborators

University of South Florida

National Ataxia Foundation

Bob Allison Ataxia Research Center (BAARC)

Pfizer

Investigators

Principal Investigator:

Theresa Zesiewicz, MD

University of South Florida

More Information

No publications provided

Responsible Party:

Theresa Zesiewicz, Professor of Neurology, University of South Florida